An observational study in Neurofibromatosis Type 1, sponsored by AstraZeneca. Recruiting at 9 sites in Egypt. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by AstraZeneca · Observational
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Among 12 studies in Neurofibromatosis 1 by AstraZeneca — 3 open to participants now, 8 under way, 1 of them phase 3. Neurofibromatosis 1 · AstraZeneca.
Among the 12 studies in Neurofibromatosis 1 by AstraZeneca, this one is the only one that started in 2025, is one of 5 observational ones, and was the 11th registered.
176 studies on the registry are indexed under Neurofibromatosis 1; 43 are open to participants now.
Browse Neurofibromatosis 1 studies →3 of the 12 studies in Neurofibromatosis 1 by AstraZeneca are open now, in 3 countries. Recruiting first, the newest first:
AstraZeneca · Sites in China
Ages 18+
AstraZeneca · Sites in Korea, Republic of
Ages 3–99
By country, each count listing its studies:
Of the 28 Neurofibromatosis 1 studies recruiting now, 18 (64%) set no upper age limit and 4 (14%) stop at 75 or younger; 19 take children under 18; 5 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
12 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 2 · Phase 3 1
First in 2018; this study’s year is marked. 1 started in 2025.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 200 is close to the median of 188 across 58 observational studies indexed under Neurofibromatosis 1.
Median enrollment 24 across the 7 interventional studies, 200 across the 5 observational. Of the 3 that completed or were terminated, 0 (0%) have results posted.
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AstraZeneca is the lead sponsor of 3,502 studies on the registry; 273 are open to participants now.
Of its 368 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
1 of the studies in Neurofibromatosis 1 by AstraZeneca that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05309668AstraZenecaPharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN | 2027-11-30 |
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Among the 12 studies in Neurofibromatosis 1 by AstraZeneca, from their registry updates:
This tab as a report to print or save as PDF → With an account.
Neurofibromatosis type 1 (NF1), a genetic disorder, results from NF1 gene mutations with nearly complete penetrance (1). NF1 is considered common as a rare disease; it has a birth incidence of approximately one every 3000 and a prevalence of one case every 3000-6000 individuals.
Patients with NF1 present lifelong phenotypic variabilities, including those mentioned in the National Institutes of Health (NIH) diagnostic criteria: multiple cafe-au-lait macules, freckling, Lisch nodules, cutaneous neurofibromas, plexiform neurofibromas (PNs), optic pathway gliomas (OPG), and osseous lesions (1). Regarding PNs, they are present in about 30-50% of NF1 patients with deeper growth along internal nerve plexus cranial or large peripheral nerve sheaths, compared to cutaneous neurofibromas.
NF1 clinical expression is unpredictable, age-related, and varies among patients; additionally, as a tumor predisposition disorder, it is associated with neoplastic complications that impair health-related quality of life (QoL). Thus, it is essential to gather data about the natural history of the disease to understand its burden on patients with NF1 and those who develop PN.
Besides that, NF1 prevalence and patients' clinical characteristics are not well recognized in Egypt, and full surgical resection of PN is often challenging due to its invasive nature, location, and size. Accordingly, this is a disease registry to collect data about patients with NF1, both pediatrics and adults. And to understand the natural history of this disorder in Egypt over the past 14 years in real-world settings. For patients with NF1, with or without PNs, we aim to understand their treatment patterns and explore clinical and nonclinical factors influencing targeted outcomes.
Patients' records will be eligible for study inclusion if the NF1 diagnosis date is between 01-Jan-2010 and 31-Dec-2023.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
A. Male or female patients of any age at index date (first diagnosis of NF1 and/or PN).
B. Have been diagnosed with NF1 according to the NIH Consensus Development Conference diagnostic criteria or the revised criteria between 01-Jan- 2010 and 31-December-2023.
Exclusion Criteria:
A. Missing NF1 diagnosis data in their medical record.
Prevalence of patients with Neurofibromatosis type 1-Plexiform Neurofibromas
Prevalence of patients with NF1-PN from total NF1 population.
Time frame: 13 Years
Demographics
Age at first diagnosis of NF1
Time frame: 13 Years
Clinical characteristics
Medical and surgical history
Time frame: 13 Years
Patient management
NF1-related medication history
Time frame: 13 Years
Patient progression
describe change in the growth of PN
Time frame: 13 Years
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Time frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Data sharing details ↗ · vivli.org
No publications or documents are linked to this record.
Started Nov 2025 (actual). Primary completion is estimated for Sep 2026, 10 months after the start.
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